Prader-Willi Syndrome Association | USA’s Post

AMX0035 Shows Promise for Treating Wolfram Syndrome 🌟🧬 Amylyx Pharmaceuticals' AMX0035 demonstrates potential in treating Wolfram syndrome, a rare neurodegenerative disease. Initial clinical data reveal improvements in patient outcomes, marking a significant step forward in addressing this debilitating condition. Continued research aims to validate these findings and expand therapeutic options for Wolfram syndrome patients. To read the full news, visit 👉 https://lnkd.in/dfKyj8uD Stay updated with the latest in pharmaceutical advancements at https://lnkd.in/dKhZfHxi #PharmaNow #pharmanews #healthcareinnovation #globalpharma #wolframsyndrome #rarediseases #clinicaltrials #AmylyxPharmaceuticals

#News: Gilead Sciences Sciences (GILD.O), agreed to acquire CymaBay Therapeutics (CBAY.O), for $4.3 billion on Monday, adding a treatment for chronic #liverdisease to its portfolio. Shares of #CymaBay were halted ahead of the announcement in premarket hours. #Gilead will pay $32.50 in cash for each share of CymaBay for the drug developer, representing a premium of 26.5% to Friday's close. The deal will give Gilead access to CymaBay's #leaddrugcandidate called #seladelpar for the treatment of primary #biliarycholangitis, a type of chronic inflammatory liver disease. Read more from Reuters 👇🏽 https://lnkd.in/eppcNZzu

Exploring the advantages of multi-target therapeutics, the focus is on minimizing drug dosage while maximizing effectiveness, a critical approach in treating complex diseases at Canurta. #innovation #research #therapeutics

SpringWorks Therapeutics announced that the Company has initiated a rolling submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for mirdametinib, an investigational MEK inhibitor, in pediatric and adult patients with neurofibromatosis type 1-associated plexiform neurofibromas (NF1-PN). The NDA submission includes data from the Phase 2b ReNeu trial. The primary endpoint was confirmed objective response rate (ORR). The BICR-confirmed objective response rate was 52% in pediatric patients and 41% in adult patients. Mirdametinib treatment showed deep and durable responses and demonstrated significant improvements in key secondary patient-reported outcome measures. Chemenu has been working to develop more compounds for drug discovery. Here come the related building blocks we can provide: https://lnkd.in/gqi63U3g 👇 #Chemenu #SpringWorks #NDA #FDA #mirdametinib #MEKinhibitor #NF1-PN #Phase2bReNeutrial #ORR #drugdiscovery #buildingblocks

𝐅𝐃𝐀 𝐆𝐫𝐚𝐧𝐭𝐬 𝐅𝐚𝐬𝐭 𝐓𝐫𝐚𝐜𝐤 𝐃𝐞𝐬𝐢𝐠𝐧𝐚𝐭𝐢𝐨𝐧 𝐭𝐨 𝐀𝐫𝐭𝐡𝐫𝐨𝐬𝐢 𝐓𝐡𝐞𝐫𝐚𝐩𝐞𝐮𝐭𝐢𝐜𝐬’ 𝐀𝐑𝟖𝟖𝟐 𝐟𝐨𝐫 𝐆𝐨𝐮𝐭 𝐓𝐫𝐞𝐚𝐭𝐦𝐞𝐧𝐭 Arthrosi Therapeutics Arthrosi Therapeutics, Inc. announced that the FDA has granted Fast Track Designation to AR882, a next-generation URAT1 inhibitor, for the treatment of clinically visible tophi in gout patients. This significant milestone underscores the FDA's recognition of the serious and disabling nature of gout in patients with visible tophi. The Fast Track Designation reflects the potential of AR882 to meet this critical medical need. With our pivotal Phase 3 clinical program now underway, we are committed to working closely with the FDA to accelerate the development of AR882 and bring it to patients as quickly as possible. #ArthrosiTherapeutics #GoutTreatment #URAT1Inhibitor #FDAFastTrack #Biotech #Pharma #ClinicalTrials #HealthcareInnovation #GoutResearch #ArthritisTreatment

CMS PHARMA DRUG PRICE NEGOTIATIONS UPDATE: CMS offers Pharma more time and opportunities to submit counteroffers during price talks that may finalize in 2025. In August, the federal government announced the lower negotiated prices for 10 medications, which include therapies for Type 2 diabetes, blood clots and heart failure. The new prices — which are estimated to reduce healthcare costs by $7.5 billion — will go into effect Jan. 1, 2026. For the next phase, CMS will select up to 15 medications to undergo negotiations for lower list prices to take effect Jan. 1, 2027. Multiple cancer therapies are predicted to be chosen, and CMS will announce the selected drugs by February. In contrast to the first round of negotiations, CMS will hold up to 15 patient-focused roundtables and one town hall that weighs the clinical considerations, according to a 313-page final guidance published Oct. 2nd. #CMS #PHARMA #NEGOTIATIONS #UPDATE #AHCA

Today Cadrenal Therapeutics, Inc. (Nasdaq: CVKD) shared an exciting analysis of a clinical trial conducted by Abbott supporting our belief that Cadrenal's lead drug candidate tecarfarin could be an important next-generation anticoagulant for patients with LVADs. Quoted in the news release lead investigator of the trial, Dr. Mandeep Mehra, states, "…Tecarfarin could potentially be an important therapy for patients with LVADs who all require chronic anti-coagulation since it does not get affected by drug-drug interactions or changes in kidney function like warfarin and deserves further study." Read more: https://lnkd.in/gypE4XZc   #cadrenal #nasdaq #cvkd #therapeutics #tecarfarin Quang Pham Matthew Szot

FDA Awards Orphan Drug Status to Disc’s Treatment for PV The FDA has granted orphan drug designation to DISC-3405, an investigational therapy for polycythemia vera (PV), a rare hematologic condition. PV poses challenges and risks, and there is a need for innovative treatments. DISC-3405, developed by Disc Medicine, aims to modulate iron homeostasis and has shown promise in preclinical studies. The drug is currently in Phase I clinical trials. Orphan drug designation provides benefits and incentives for developing treatments for rare diseases. This milestone represents hope for PV patients and the broader community. The FDA's support for innovative therapies is crucial. The journey of DISC-3405 exemplifies the challenges and opportunities in developing treatments for rare conditions. This designation is a significant step forward in fighting PV and other rare hematologic conditions. For more details please click the link! https://lnkd.in/dJwE_ngb #marketaccess #reimbursement #pricing #hta #heor #healtheconomics #medicaldevices #pharmaceutical

Travere Therapeutics achieved a milestone with the FDA’s full approval of #Filspari for immunoglobulin A nephropathy (#IgAN), removing prior restrictions and expanding its potential market. As the first non-immunosuppressive treatment for IgAN, Filspari is set to drive significant revenue, with 2024 sales forecast at $118 million, based on Visible Alpha consensus. While Filspari's expansion is promising, it carries safety warnings limiting distribution. Travere’s overall revenue is expected to rebound by +54% in 2024, supported by Filspari’s growth and ongoing trials for additional kidney conditions. Read more: https://lnkd.in/d2NrTSiQ

55 Novel Drug Approvals for 2023 🔥 A WRAP-UP 🔥 As we say goodbye to 2023, let's look back at another year of RELENTLESS drug discovery! check them out!🙌 👉 55 new drugs approved by the FDA's Center for Drug Evaluation (CDER), up from 37 drug approvals in 2022. 👉 32 of these NMEs can be classified as small molecules. Multiple drug modalities – SMs, mAbs, bispecifics, enzyme replacement therapies, ASOs, PET agents, siRNA and bark extracts even. 👉 16 oncology, 6 neuro, 6 I&I, 5 infectious disease, 5 metabolic disorder drugs. #medicine #FDAapproval #drugdiscovery #drugdevelopment #newdrugs #2023throwback #drugapproval Image from Chris De SaviChris De Savi